St. Jude Children’s Research Hospital is offering a stable cell line for producing a gene therapy vector for treating Wiskott-Aldrich Syndrome (WAS).The WAS vector uses the same lentiviral backbone as a vector made by St. Jude for treating X-linked severe combined immunodeficiency disease (“XSCID”), which is currently being tested in a phase I clinical trial.
Gene therapy, Wiskott, Aldrich, transplant, lentiviral
Granted Patents or Published Applications
Related Scientific References
Singh, S. et al., “Safe and Effective Gene Therapy for Murine Wiskott-Aldrich Syndrome Using an Insulated Lentiviral Vector”, Molecular Therapy 4: 1-16 (Dec. 18, 2016).
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